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Congenital, Hereditary, and Neonatal Diseases and Abnormalities

  • Long-Term Study on the Safety and Effects of RP-L102 Infusion for Patients with Fanconi Anemia Subtype A

    Recruiting

    1 1 1
    Investigated Diseases:
    • Congenital aplastic anaemia
    Investigated Drugs:
    • Autologous Cd34+Enriched Cells From Patients With Fanconi Anemia Subtype A Transduced Ex Vivo With Lentiviral Vector Carrying The Fanconi Anemia Complementation Group A Gene
    Spain

  • Study on Long-term Effects and Safety of Odevixibat in Children with Biliary Atresia

    Recruiting

    3 1 1
    Investigated Diseases:
    • Congenital absence of bile ducts
    Investigated Drugs:
    • Odevixibat
    Belgium France Germany Hungary Italy The Netherlands +2

  • Study on the Safety and Effects of UX701 Gene Therapy for Adults with Wilson Disease

    Recruiting

    2 1 1 1
    Investigated Drugs:
    • Adeno-Associated Viral Vector Serotype 9 Encoding Human Atp7B
    • Eculizumab
    • Rituximab
    Denmark France Germany Italy Portugal Spain

  • Study on Hydrochlorothiazide and Tolvaptan for Patients with Autosomal Dominant Polycystic Kidney Disease (ADPKD)

    Recruiting

    3 1 1
    Investigated Diseases:
    • Congenital cystic kidney disease
    Investigated Drugs:
    • Hydrochlorothiazide
    • Tolvaptan
    Austria Belgium France Germany The Netherlands Spain

  • Study of Microdystrophin (GNT0004) Gene Therapy for Duchenne Muscular Dystrophy in Boys Who Can Walk

    Recruiting

    2 1 1
    Investigated Drugs:
    • Adeno-Associated Viral Vector Serotype 8 Containing The Human Md1 Gene
    • Calcium Chloride Dihydrate
    • Potassium Chloride
    Belgium France Spain

  • Finding the Right Dose of Dobutamine for Very Preterm Infants with Heart and Blood Flow Issues

    Recruiting

    1 1 1 1
    Investigated Diseases:
    • Haemodynamic instability
    Investigated Drugs:
    • Dobutamine
    Spain
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The European Clinical Trials Information Network is transforming access to clinical trial information for patients across Europe. By overcoming language barriers, our platform allows everyone, regardless of their native language, to easily find relevant and comprehensible clinical trial data. This innovative service connects patients to cutting-edge treatments, creating a unified European community where health and progress transcend borders.

This service is not affiliated with the European Commission, the EMA, or the official CTIS system. Most information comes from publicly available international clinical-trial registries, supplemented by data from academic centres, national regulators, and commercial sponsors. We aim to keep all content accurate and up to date.

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